Jay Backstrom; President, Chief Executive Officer, Director; Scholar Rock Holding Corp
Good morning and welcome to Scholar Rock's fourth quarter of financial results and business update call. (Operator Instructions)
Please note this event is being recorded. Before we begin, I'd like to point out that we'll be making various statements about Scholar Rock's expectations, plans, and prospects that constitute forward-looking statements for the purposes of the Safe Harbor provisions under the Private Securities Litigation Reform Act of 1995. Any forward-looking statements represent our views only as of today and should not be relied upon as representing our views as of any future date.
I encourage you to go to the Investors and Media section of our website to find our most up-to-date SEC statements and filings. A recording of today's event will also be available on our website should you want to re-watch at a later date.
I will now turn the conference over to Jay Backstrom, President and CEO of Scholar Rock. Mr. Jay, please go ahead.
Well, thank you, operator. Good morning, and welcome, everyone, and thank you for joining our fourth-quarter 2024 business update. We had an outstanding year in 2024, and we're off to a great start to 2025.
For today's call, I'll start by providing a review of the SAPPHIRE results and our progress toward our regulatory milestones. Tracey Sacco, our Chief Commercial Officer, will share the terrific progress we're making with our commercial preparation and our planning for a 2025 launch as we're working with a sense of urgency to serve those living with SMA globally, starting with the US.
And then I'll follow with a review of our innovative myostatin platform and our ambition to transform the current GLP-1 treatment paradigm for weight management. Following our prepared remarks, Tracy and I will be joined by Ted Myles, Chief Operating Officer and Chief Financial Officer; and Mo Qatanani, our Chief Scientific Officer, for the Q&A portion of today's call.
Moving to slide 4. With our success in 2024, the stage is set for what will be a transformative year for Scholar Rock. And as I said, we are working with a sense of urgency to bring a apitegromab to those living with SMA, and we continue to hit our milestones on time or ahead of schedule.
Starting with our regulatory applications for a apitegromab and SMA, we submitted the in the US in January, and we remain on track to submit the MAA in the EU in March. We look forward to sharing the SAPPHIRE data at the Muscular Dystrophy Association's annual meeting in Dallas on March 19th, where this data will be featured as an oral presentation.
We will also be sharing our work with the murine version of a apitegromab in a non-clinical model of DMD, setting the stage for us to expand the development of a apitegromab into other neuromuscular indications. We also continue to make progress on our goal to expand treatment to even the youngest of those with SMA, and we're on track to open the OPAL study for those under two years of age in the third quarter.
For our cardiometabolic program, we remain on track to share the top-line data from EMBRAZE and Q2 with the filing of the IMD for SRK439, our highly selective anti-myostatin designed for cardiometabolic indications, in the third quarter.
Let's turn to slide 5. Before I hand over to Tracey, I want to review the positive top-line results for SAPPHIRE, our registration study with the apitegromab, the only muscle-targeted therapy with clinical success in a pivotal study in SMA, which has the potential to transform the standard of care.
Apitegromab plus standard of care delivered gains of 1.8 points improvement compared to placebo plus standard of care, as measured by the gold standard SMA-specific Hammersmith functional motor scale at week 52, gains that were both clinically meaningful and statistically significant.
Importantly, there was consistency across age groups 2 through 21 in a broad SMA population. In addition, 30% achieved an additional 3 point or greater improvement in their Hammersmith scores when compared to placebo plus an SMN therapy, where only 12.5% achieved this same high bar. Further, as shown in the graph, displaying motor function over the 52-week treatment period, those receiving a apitegromab achieved a gain in function, while those receiving an SMN therapy alone lost function over this 52-week period.
With respect to safety, the safety was consistent with the TOPAZ data, with over 95% rolling over and remaining in the long-term follow-up study, adding to our experience in over 200 patients, and we remain the only muscle-targeted therapy that has over four years of clinical experience in SMA.
Together, the data support an overall favorable benefit-risk with the potential to shift the treatment paradigm and usher in a new standard of care to include apitegromab, a muscle-targeted therapy, with an SMN-directed therapy, as part of the treatment regimen for SMA.
I will now turn the call over to Tracey, our Chief Commercial Officer, to provide an update on our commercial preparations. Tracey?
Tracey Sacco
Thank you, Jay. Moving to slide 7, despite successes in treating the motor neuron over the past eight years, progressive muscle weakness continues to be a critical unmet need in SMA, and there is currently no approved muscle-targeted therapy to treat this neuromuscular disease.
Patients and caregivers have described to us the debilitating impacts of progressive muscle weakness and how it significantly detracts from their independence and their ability to perform basic daily activities, like eating, dressing, getting in and out of bed or their car, brushing teeth, climbing stairs, and using the bathroom.
This is why 97% of patients surveyed by Cure SMA identified improving muscle strength as an important need that they want to see from a new SMA treatment. From our own market research, we know that more than 80% of treating neurologists agree that preserving muscle should start as early as possible in SMA. The SMA community is collectively calling for more to treat this relentless progression of muscle weakness and improve motor function in SMA.
Next slide. Today, roughly two-thirds of the 10,000 people living with SMA in the US and 35,000 individuals globally have already received an SMN-targeted therapy, yet we see from the data that Jay just presented that despite these effective therapies, progressive muscle weakness still robs these individuals of their ability to function over time. This is why 74% of neurologists recognize that in the future, a combination of modalities to target the motor neuron and the muscle will be necessary to treat SMA.
The future of SMA will be to directly treat both the muscle and the motor neuron to provide the best outcomes for patients. Apitegromab is a potential first-approved muscle-targeted therapy in SMA is leading the transformation of SMA care.
Next slide. Scholar Rock has been at the forefront of preparing the market for this potential new treatment paradigm in SMA. Last year, we introduced Life Takes Muscle, the first muscle-focused SMA disease education campaign that amplifies what we've been hearing from patients and caregivers for years about the devastating impacts of progressive muscle weakness. Life Takes Muscle has resonated with both the patient and HCP communities and led to continued strong engagement with Scholar Rock.
Additionally, our MSL team has also been meeting with the leading Cure SMA centers and engaging with top treating neurologists and care teams to educate on our Phase III SAPPHIRE top-line data. And Scholar Rock continues to partner and learn from our patient advocacy partners who have been so effective already in helping to bring new treatments to the SMA community.
Next slide. We're doing all this with a team of experienced professionals with deep rare disease and launch experience, including prior SMA launches, and we're continuing to be a magnet to attract top commercial talent as we build out our team.
Next slide. 2025 is an important year for Scholar Rock, and we are positioned for a successful launch. We'll continue to build on our stakeholder engagement and education, and this quarter have initiated outreach to top US commercial and federal payers ahead of our potential Q4 launch. We will also be scaling our customer-facing team of roughly 50 sales, reimbursement, and patient support personnel in late Q2 and early Q3 to be hired and onboarded ahead of our potential launch.
And finally, Scholar Rock is building out our patient services offering and preparing to offer home infusion as an option at launch to provide excellent support and optionality to patients and treaters alike. We have been laying the groundwork for years, and now we're putting the broader team and infrastructure in place for a successful US launch to meet a critical need in the fight to continue to improve the lives of those living with SMA.
I'll now turn the call back to Jay.
Jay Backstrom
Well, thank you, Tracey.
And now turning to our cardiometabolic programs and moving to slide 13. It's impressive to see the impact of the innovation from our industry and how the approved GLP-1 receptor agonists have transformed the management of weight loss. Obesity is a top global public health issue, and the potential public health impact of these highly effective weight loss therapies have been incredible across a number of chronic conditions, including diabetes, sleep apnea, and cardiovascular disease. With an estimated 40 million projected to receive treatment GLP-1 receptor agonists, the current market is anticipated to generate over $100 billion in sales.
Moving to slide 14. With the extraordinary amount of weight loss seen with the current GLP-1 receptor therapies, there's also a substantial amount of lean muscle that is also lost, ranging from [12% to 14%]. With the widespread use of these therapies, there's an increased awareness of the associated weakness and reduced strength that accompanies this loss of lean muscle mass and of the significant weight regain that occurs when stopping therapy, with disproportionately more fat being regained than lean mass, leading to a worse body composition and leading to unhealthy weight gain.
Beyond strength and mobility, muscle is the main metabolic organ and plays a significant role in energy metabolism, increasing basal metabolic rate, glucose uptake, and improving insulin sensitivity. Given the critical role that muscle plays in overall health, we believe our highly selective approach to blocking myostatin when added to the GLP-1 receptor agonists can enhance their profile by reducing the loss of lean muscle mass without introducing additional toxicity, leading to healthy and sustainable weight management.
Moving to slide 15. To illustrate what can be expected from tirzepatide, this figure is from the tirzepatide SURMOUNT-1 study and illustrates the percent change in body weight over time. The area shaded in green represents the first 20 weeks of the study, the time period corresponding to the efficacy assessment and embrace, our randomized Phase II group of concept study with tirzepatide.
As can be seen, even by week 24, there's an impressive amount of weight loss of 14% to 16% of body weight that continues to deepen over the course of 72 weeks. Approximately 25% of this weight loss is lean mass, an area that we believe we can address through our highly selective approach to blocking myostatin, the master regulator of skeletal muscle. By preserving lean mass and reducing the amount of lean muscle mass lost, it can lead to healthier and sustainable weight loss and potentially change the treatment paradigm for weight loss management.
Now, moving to slide 16. So what does preserving lean muscle mass mean with respect to clinical benefit and what amount is needed to have a meaningful impact? What insights can be gained from a study of healthy young men who were confined to strict bed rest for one week and lost about a kilogram of lean mass as assessed by DEXA scans?
Remarkably, the loss of just one kilogram of lean mass resulted in significant decrease in leg strength, exercise capacity, and equally importantly, in insulin sensitivity compared to baseline. Considering that adults over 60 begin to lose 1% of lean mass per year, with a 10% to 15% decrease in strength per decade, the additive effect of loss of lean mass associated with GLP-1 receptor therapy in this age group and other subsets can be profound.
Now, moving to slide 17. With respect to EMBRAZE, we had several key goals in mind when we designed EMBRAZE, a randomized Phase II proof-of-concept study comparing tirzepatide plus epidemiopathy to tirzepatide plus placebo in obese or overweight adults.
First, with respect to preserving lean muscle mass, we believe we can reduce the amount of loss as measured by DEXA scans at week 24 with our highly selective approach to blocking myostatin. We selected week 24 as the time point to assess since this was the steepest portion of the weight loss curve of tirzepatide, recognizing that we'll need to assess at week 52 for our SRK-439 program.
As a proof-of-concept study, we're looking for trends in the magnitude of effect to help shape our thinking regarding clinical meaningful improvement. Extrapolating from the study in young men, assuming a loss of lean mass between 5 and -- 4 to 5 kilograms, by reducing the amount of lean mass lost by 20% to 40%, we can preserve 1 to 2 kilograms of lean mass, which we believe will translate into clinical meaningful benefit since every kilogram of lean muscle mass matters.
Second, based on our selective approach, one of our goals was to demonstrate that we can save the combined tirzepatide and not introduce any additive toxicity that can be associated with blocking other TGF beta-ligand such as acid. Third, with respect to weight loss of week 24, we are expecting to see comparable weight loss at this time.
Fourth, based on our non-clinical data, we believe we can blunt the fat regain associated with stopping tirzepatide and have built in an additional DEXA scan performed eight weeks after stopping treatment to assess this important question. These data will not be part of the top-line results that we look forward to sharing next quarter.
Fifth, as approved for consult study, we also included exploratory endpoints such as hemoglobin A1C and functional measures, but we did not power or enrich the study with subsets of patients to show statistical significance for these exploratory endpoints.
And then finally, an important key goal is for us to gain experience in the setting of obesity. We'll have the opportunity for detailed review of all the EMBRAZE subject data to identify subgroups and to help inform the clinical development of SRK-439, a highly selective anti-myostatin designed for cardiometabolic disorders. We've already shown our ability to deliver EMBRAZE ahead of schedule, and this experience provides us with great momentum for SRK-439 program.
Moving to slide 18, and with respect to SRK-439, we are very excited with the data that's been generated to date in our non-clinical program demonstrating that we can preserve lean mass with further reduction in fat mass, we can improve metabolic parameters such as blood glucose, we've seen increase in lean mass gain and once the fat, regain of fat mass after stopping a GLP-1 receptor agonist therapy, and we've demonstrated greater potency in a direct comparison with anti-ACTRII antibody.
Overall, the data suggests the best-in-class profile for SRK-439, which has been featured at key scientific conferences in 2024, and we look forward to bringing SRK-439 to clinic later this year.
Moving to slide 19, in summary, to address the important emerging problem of loss of lean mass associated with weight loss therapies, we've taken a parallel path with our innovative industry-leading portfolio of highly selective anti-myostatin therapies by conducting a proof of study, proof-of-concept study with apitegromab and EMBRAZE, and at the same time advancing SRK-439 to IND, our highly selective novel anti-myostatin designed specifically for cardiometabolic indications with the ambition to transform the current GLP-1 receptor agonist treatment therapy.
This strategy has resulted in two important milestones in 2025, and we look forward to the readout of the top-line data from EMBRAZE next quarter and the filing of the IND for SRK-439 in Q3.
In closing, and moving to slide 21, 2025 will be a transformative year for Scholar Rock. We are off to a great start. We've submitted the BLA for apitegromab in the US in January. We're on track to submit the MAA in the EU in March.
We were well on our way in our preparations to bring apitegromab, a muscle-targeted therapy, a potential new treatment option for those living with SMA globally, starting in the EU in Q4, with EU to follow in 2026, as we continue to work to expand our reach to the youngest of those with SMA and to other rare neuromuscular disorders. We are on the threshold of establishing a neuromuscular franchise, starting with SMA.
We look forward to reporting on our progress toward achieving our 2025 key milestones as we drive to commercialize and expand the development of apitegromab and advance our cardiometabolic programs. Overall, a very, very exciting year for Scholar Rock. And operator, that closes the prepared remarks. We'll now open up the call for questions.
Operator
(Operator Instructions) Allison Bratzel, Piper Sandler.
Allison Bratzel
Hi, good morning guys, and thanks for taking the questions. Maybe one on SMA just with Sapphire data being presented next month at MDA. Can you just discuss your sense of what additional data points are going to be most meaningful to patients and docs when you present that full data just maybe help frame that update for us and then one on obesity.
Just maybe help us understand what you need to see on the primary endpoint to be confident in taking 439 forward. I think in the prepared marks, you discussed the 20 to 40% improvement in lien mass as being clinically meaningful just did I hear that right? And then, what are you hoping to see on, additional end points like A1C and body composition just just curious to get your thoughts there.
Thank you.
Jay Backstrom
Yeah, good, Allie. Hey, good morning. This is Jay. So first starting the sapphire, we're really excited to have the opportunity for those data to be presented at a congress and particularly the MDA conference where there's a lot of interest and a lot of investigators who have a chance to see the data beyond what we could share on the top line results. And as we shared previously, I think what's really important is the overall consistency and impact of the totality of the data. We've shared, we've got effects across age groups, and we're going to share additional endpoints and you'll see again I think there'll be interested in seeing the consistency and the impact we have across those additional endpoints. So I think that'll be a really nice opportunity to really underscore that we really are on the threshold of bringing, I think, a really nice new effective therapy to change standard of care and that.
And then with respect to the bras and 439, right, I said we have a very unique opportunity. We've got a really, I think, highly innovative platform of antimyostatin therapies. We took full advantage of being in clinic to study embrace.
As we're looking, I TRY to put some context around, what is clinically meaningful with lean mass loss, because that's a question that comes to us constantly. I do think there's insights gained from that healthy that study of young men who were confined to bed rest that lost a kilogram. Because that was a significant amount of strength loss even in a young healthy person, right? So as we think about going forward, that 1 kg, that translates to about a 20% effect. We can double that, that's 2 kg. So I think we're in that range of signal seeking that will give us insight that will allow us to continue to really think deeply about how we develop 439. But I want to make a caveat. We're studying this at week 24 on the steepest part of the slope of the curve of his appetite. And of course we'll do a 52 week study as we get into 439. So there's a potential for that effect only mass to deepen over time. So we're looking at a snapshot at week 24. I think we can show a nice effect. And in that range of 20 to 40% where we can think we have a meaningful incremental benefit, I think we have a really clear go signal to move forward 439. But we'll also be developing 439. We'll have to establish its own, dose, etc. So, lots to come with this total program, but very exciting time for us.
Allison Bratzel
Excellent thank you.
Operator
Michael Yee, Jefferies.
Michael Yee
Hey guys, thanks for the question. Good morning. Just following up on thoughts around the obesity readout in the 2nd quarter, I appreciate that the expectation is that you should preserve lean muscle. I was just wondering about how you guys. Thinking about the biology or the effects of what would happen on the actual weight loss, there shouldn't be any material change, but I'm just wondering if there could actually be maybe more weight loss because of the metabolic effects of myostatin and building muscle, or there could actually be headline a little bit less weight loss just because you're actually preserving the muscle. So just thinking about the bio there certainly in the 1st 24 weeks and how to put that in the context of preserving muscle.
Thank you.
Jay Backstrom
Yeah, Mike, thanks for the question. I TRY to frame that in a bit in the prepared remarks. So for week 24, given the magnitude of weight loss that's already occurring with his appetite, really don't expect at that time point to see, we think the weight loss will be comparable between the two loss, recognizing that there's a potential with increasing muscles to potentially affect weight, but the magnitude of the weight loss which his appetite is probably going to make that a wash. And so we think it'll be comparable at week 24. But to add to your point, now that's at week 24.
If we look at effects over time and the potential effects on basal metabolic rate and maintaining, there absolutely is a potential to see some additional incremental weight loss over a period of time on treatment, but I want to set the expectations for week 24 we're expecting to.
Got it thank you very much.
Operator
Tessa Romero, JPMorgan.
Tessa Romero
Hi, good morning, Jay and team. Thanks so much for taking our questions. I look forward to seeing you guys at MDA. So what is the right way to think about the exploration of additional neuromuscular indications where a Pittagram app could have potential here? What framework? You give us with respect to those decisions. In other words, how you will best allocate capital while also ensuring proper investment in the launches for SMA and also the cadence and timelines with which you could or would start these clinical studies. Thanks so much.
Jay Backstrom
Yes, Tess, thank you for the question. Very important question. We're going through that work right now, as I indicated at the JPMorgan conference, and with the success of a pittogram of an SMA.
I mean, I almost feel obligated for us to take a look at the adjacent neuromuscular indications to see if in fact we can have an opportunity to impact and improve lives for those that have other diseases like BMD, FSHD, Beckers, and even ALS. So what we're showing at MDA is a bit of the work and thinking behind our approach to this.
I'm very keen on translational models to the extent that they can give us insight, and I feel very fortunate with Mo and his team to be able to do these models and so we're going to start showcasing how we're beginning to think about, for example, DMV a little bit with that non-clinical data. We're actively engaging experts across. These indications to really begin to think about how we can take a look at both not only the opportunity in terms of the unmet need, but the probability of technical success and can we understand and enrich a patient subset of patients that we can really show meaningful benefit. We're doing all of that work and as we put all that together, that'll set the framework as we think about further investment in those indications, which as you can imagine is going to be. Function of technical success, unmet needs, potential opportunities, etc. So we're doing all that work now over the course of, frankly, we started that work even last year. So that work continues and as we get closer to that, we'll certainly share how we think with respect to our ability to to do this in addition to doing our launch. We have a clinical team that really has demonstrated their ability to execute clinical trials. Sapphire is finished. We have the ongoing study in the long term follow up, but we're positioned to be able to take on an additional clinical study, if not two, but certainly one as we think about going forward. And again, what I tried to share at JPMorgan was if you think about what we're trying to build here and the value we're trying to create at Scholarrock, it starts with SMA. And as we said, we think that there's a potential $2 billion plus opportunity for us in SMA alone, considering what we believe will be a paradigm shift in treatment. And then if you start to build around these additional neuromuscular indications, we're we're on the threshold assuming execution and success, to build a multi-billion dollar neuromuscular franchise, and I think at the end of the day if we do that properly, we'll definitely increase shareholder value, but importantly extend our reach to those in need.
Thank you.
Operator
Marc Frahm, TD Cowen.
Marc Frahm
Hi, this is Alex. I'm from Mark. Thanks so much for taking my question.
So the FDA recently issued a draft guidance on obesity clinical trials which appeared to reinforce the agency's focus on BMI and overall weight loss rather than shifting the focus to body composition changes and preservation of lean muscle mass. What do you view as the potential implications here for your cardio metabolic program program, namely 439. Thanks.
Jay Backstrom
Yeah, it's a good question. We're obviously following this with great intent and seeing and looking carefully at FDA's updated guidance. From my perspective, kind of watching FDA over the years, they tend to move slowly. They tend not to go as fast as the field is trying to get them to go in terms of end points. So we were not surprised that they retained BMI.
The things that we took of interest though from that guidance, which I think further reinforces why I think we're actually on the right track here. Is that now very clearly in that guidance they're asking all sponsors to include within their clinical development, the very least a subset of their patients to understand the amount of lean mass loss on their treatment.
So it's recognizing that the loss of lean mass has the potential to not be a good thing. So let's assess that at baseline and understand it across programs. So I think that further reinforces the question around lean maps.
They clearly indicated in that guidance of if there's an interest in body body composition, come talk to us. So that to me is a bit foreshadowing where they may be tilting but clearly that's not within the guidance now. The body composition matters and the field is declaring that as a really important event or end point. So I think that's something that we'll continue to follow with great attention, but also which I believe will affect positively if in fact we preserve we met. So we'll have a nice impact on that. But then they also said what is very clear is that if you're doing a combination program then you need to demonstrate some additional benefit from our drug, for example, SRK 439. And what I was trying to showcase in today's discussion to bring further clarity to that, but what I said pretty consistently across our entry into this is that those end points that could be regulatory approvable endpoints are metabolic parameters such as further reduction in hemoglobin A1C.
Clearly, there's a potential for that given the role of muscle. And there's clearly an opportunity to find a way to demonstrate that losing this amount of lean mass really results in functional loss that's significant, and we have the potential within a subgroup of patients to show those functional measures and improvement, which ultimately I think will be sustainable. And then there's other ways that we can look at how we can manage through mitigating weight regain, maintenance approaches, etc. We have a lot of ideas of how to move this forward when we get into clinic. But I think the guidance to come back to it are consistent, but I think also we're pointing the way, and I feel very good about the fact that we're in this space.
Great, thank you.
Operator
Gary Nachman, Raymond James.
Gary Nachman
Thanks and good morning. So yeah, back on Embrace, if you hit the 1 to 2 kg of lee mass preservation with epigramab, then how would you expect 439 to perform based on that? So how much better could it potentially be than a grim based on the preclinical data that you've seen since the space is getting a lot more competitive, so trying to think from, a commercial marketing perspective as well.
And then just with SMA maybe just a bit more on how the initial payer discussions are going and any update on how you're thinking about pricing as a combination drug, how that's going to be received in the market.
Jay Backstrom
Thanks.
Yeah, Gary, thanks for the question. I'll start with the 439 question and then you know I'll turn to Tracy to to to address the question on our interaction so support with payers.
It's interesting. It's like having two children and you're going to say which one is better, right? I mean, I think what I would say to you, Gary on 439, I really like the profile that we're developing. I really do. I think the work that Moe and his team are showing is this is performing beautifully. It has greater affinity for the target, and that could potentially help us certainly at least with the very much with the dosing such that we can then go in with the low dose subcu formulation. So that is a clear advantage and we're looking forward to that. Now with respect to how much different and how much better, that's a really interesting question, Gary, because what I was trying to say is if Peterroma is like signal seeking, yes, we can show this.
439 is the program and we will need to further explore with 439 how we optimize the dose of 43.
And how we begin to get some better insight into how its impact across these functional measures. So that's part of our thinking on the IMD opening studies. So it's more to come with 439 at the very least, it's going to have similar effect. I do think though it's designed to be low dose kind of subcu presentation which in this highly competitive space I think is something that we need to do. And so more to come, but we're really moving rapidly on that toward IND. And then Tracy, you want to make a comment on the payer interactions today?
Tracey Sacco
Yeah, sure, pairs have been very receptive to hearing from us, even as a new company and are very interested in learning more about our innovation as the first muscle targeted therapy and SMA.
They are familiar with SMA and recognize that there's still a continued need to treat this relentless disease, and we know today that there are already a third of lives covered of US commercial payers already have policies covering what I would call combination treatment of an SMN targeted therapy post gene therapy. So that's a reflection of the openness to address this recognition of unmet need. Ultimately, we expect US and European payers alike to enable access reflective of the value of Pitroment brings. In SMA and our data and overall profile are really strong. We have a clear added benefit across all cohorts and a highly favorable safety profile, so it sets us up for really strong discussion.
Gary Nachman
Okay, and just pricing, any latest thoughts there just typical like in line with the other SMM treatments?
Tracey Sacco
Yeah, I mean, at this point, we're not in a position to share pricing, but ultimately we expect that access to be covered reflective of the value we bring, and you, we do have a sense of what the pricing is in the estimate market today.
Okay, great.
Thank you.
Operator
Srikripa Devarakonda, Truist Securities.
Srikripa Devarakonda
Hi, this is Alex for Cripa. Thanks for taking our questions. To talk about The future development path for the muscle preservation drug to be a little specific, we know that that overall we definitely see the benefits of more muscle is better, but when it comes to the regulatory review and the specifics on the endpoint, you mentioned a couple of different avenues that that you could explore with the HbA1C and mitigate weight regain. But when it comes to the strength endpoints. We know we've seen grip or stair climb, are these end points that are on the discussion table or are there other strength related functional outcomes that come to mind as the potential options going forward for the program.
Jay Backstrom
Thanks.
Yeah, really good question. As you can imagine, we've given this a lot of thought as we think about how to then really meaningfully move 4,394 beyond just identifying the right dose. And you know what I kind of add color to your to your point, I think about once we established the dose, can envision that we would then have subgroups of patients in our proof of concept efforts to demonstrate the impact we could have on, for example, hemoglobin A1C. And in that context, we would definitely enrich for a subset of patients where we think we can meaningfully show that difference. So that, that's a very clear regulatory path forward. That's the metabolic value, the additional benefit of preserving that lean muscle. So that's one example.
And the other example that you touched on which comes immediately to mind, and we've demonstrated functional improvement in SMA, so clearly functional measures and functional improvement are interesting.
We're not wed to grip strength and stair. We're giving a lot of thought around this. I think there's a lot of insight that we're gaining from our work and our review and discussions, and we'll have a really good opportunity when we open up the IND with 439 and begin to meaningfully engage FDA in our thinking around the development program, thinking around meaningful functional measures and endpoints, and at the same time get some. Sense of how we impact those functional measures as we build the program from sad mad into those early POCs so that we'd be in a really strong position to know exactly how to run in a registration program. So that's our thinking. I feel really good about it. It's like we're, it's like stair stepping, right? I love the fact that we've got embrace coming in the IND is going in 49. We've really taken advantage of our platform. Those are both coming. Got this momentum at 439 and now we're already beginning to see what the next couple steps are. So I'm, I tell you, I can't wait to share all that with you. One step at a time though, we're driving to get the IND open. We're doing all this nice work, more to come, but I feel really good. We've got great momentum running into this ID.
Srikripa Devarakonda
Yeah, that's great.
Thank you for the call.
Operator
And I'm not showing any further questions at this time. I turn the call back over to Jay for any further remarks.
Jay Backstrom
All right, but just to close, listen, thank you for your interest. I mean, it really is an exciting time. I TRY to put energy into my voice when I do this prepared remarks. I feel like I have more energy when we get into the Q&A period, but H1stly, with this team is hustling.
Thank you for your interest, and we look forward to sharing the updates and progress over the course of the year, and I will close the call.
Operator
Thank you, ladies and gentlemen. That's conclude today's presentation. You may now disconnect and have a wonderful day.
